The R-Pharm Group of Companies has announced the registration of the drug Wiltepso (viltolarsen) in Russia. This medication is designed to treat Duchenne muscular dystrophy in patients with a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53. The drug was developed by the Japanese company Nippon Shinyaku. Two years ago, the company entered into a license agreement with R-Pharm to localize production and commercialize Wiltepso in the Russian Federation.
Duchenne muscular dystrophy (DMD) is a rare muscle disorder but it is one of the most frequent genetic conditions affecting approximately 1 in 3,500 male births worldwide. It is usually recognized between three and six years of age. DMD is characterized by weakness and wasting (atrophy) of the muscles of the pelvic area followed by the involvement of the shoulder muscles. As the disease progresses, muscle weakness and atrophy spread to affect the trunk and forearms and gradually progress to involve additional muscles of the body. In addition, the calves appear enlarged in most patients. The disease is progressive and most affected individuals require a wheelchair by the teenage years. Serious life-threatening complications may ultimately develop including disease of the heart muscle (cardiomyopathy) and breathing (respiratory) difficulties.
DMD is caused by changes (variants) of the DMD gene on the X chromosome. The gene regulates the production of a protein called dystrophin that is found in association with the inner side of the membrane of skeletal and cardiac muscle cells. Dystrophin is thought to play an important role in maintaining the membrane (sarcolemma) of muscle cells.